Section 12 · Monograph
Trofinetide
Reviewed Nov 2026FDA label
DaybueSynthetic GPE analog
- Forms
- Sol: 200mg/mL
- Dose
- Weight-based BID: 5,000mg (9 to <12kg) up to 12,000mg (50kg+), Rett syndrome age 2+
- MOA
- IGF-1-related; ↑synaptic function (Rett syndrome)
Indications
- Treatment of Rett syndrome in adults and pediatric patients 2 years of age and olderFDA-labeled
Mechanism of Action
- Synthetic analog of glycine-proline-glutamate (GPE), the N-terminal tripeptide of IGF-1; believed to enhance synaptic function and morphology
Full Dosing
Adult / usual
- Twice daily by weight: 9 to <12kg 5,000mg (25mL); 12 to <20kg 6,000mg (30mL); 20 to <35kg 8,000mg (40mL); 35 to <50kg 10,000mg (50mL); 50kg or more 12,000mg (60mL)
- Give orally or via gastrostomy tube (via the G-port for GJ tubes); with or without food
- Discard unused solution 14 days after first opening; if a dose is missed, skip it
Contraindications
- None listed in labeling
Drug Interactions
- Laxatives increase the risk of diarrhea — reduce or stop laxatives when starting
Adverse Effects
- Diarrhea (most common; can be severe — manage with hydration, adjusting laxatives, antidiarrheals and dose reduction)
- Vomiting, fever, seizure, anxiety
- Decreased appetite, fatigue and weight loss
Monitoring Parameters
- Hydration status and weight
- GI tolerance
- Seizure control
Pregnancy & Lactation
- No adequate human data; animal studies showed no embryofetal harm at the doses tested
- Unknown whether present in human milk